Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Durable responses for hemophilia A gene therapy

High K et al. ASH 2018, Abstract 487

Key clinical point: A single infusion of SPK-8011 reduced bleed and infusion rates in severe hemophilia A patients.

Major finding: The overall reduction in annualized infusion rate (AIR) and annualized bleeding rate (ABR) was 97% for each.

Study details: A phase 1/2 study of 12 patients

Disclosures: Spark Therapeutics sponsored the study. Dr. George reported equity ownership related to the University of Pennsylvania and consultancy for Pfizer.

Read the article here.


High K et al. ASH 2018, Abstract 487.